The Medical Letter on Drugs and Therapeutics
Golodirsen (Vyondys 53) for Duchenne Muscular Dystrophy
Subscribers: Log in to read full article.  Not a subscriber?  Subscribe or purchase article.
Golodirsen (Vyondys 53 — Sarepta), an antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in the ~8% of patients who have mutations of the dystrophin gene that are amenable to exon 53 skipping. It is the first drug to be approved for this indication and the third to be approved for treatment of DMD; the antisense oligonucleotide eteplirsen (Exondys 51) and the oral corticosteroid deflazacort (Emflaza) were approved earlier. ... more
The Medical Letter is a subscriber-funded nonprofit organization that publishes critical appraisals of new prescription drugs and comparative reviews of drugs for common diseases.

Would you like to read the rest of this article?  Gain access below.

Subscriptions to The Medical Letter on Drugs and Therapeutics include:
  • Print version published and mailed biweekly (26 issues/year)
  • Unlimited online access to current and past issues (1988 - present)
  • Mobile App for iOS, Android, and Kindle Fire
  • FREE online per issue CME/CE
Purchase this article:
Title: Golodirsen (Vyondys 53) for Duchenne Muscular Dystrophy
Article code: 1603d
 Electronic, downloadable article - $45
Gain access through your organization
Ask your librarian to consider an Institutional Subscription to The Medical Letter.
Recommend to your librarian